Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Mercuri E, Muntoni F, Osorio AN, et al. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. Journal of comparative effectiveness research 2020;9:341-360 Commentary by Morkous SS. Treatment with Ataluren for Duchene Muscular Dystrophy. Pediatr Neurol Briefs 2020;34:12   Abstract Aim: Strategic Targeting of Registries and […]